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Ex-vivo method to expand and maintain hematopoietic stem cells

Description:

An efficient way to improve outcomes of bone marrow and umbilical cord blood transplants to increase survival in blood cancer patients. The invention works by expanding hematopoietic stem cells in culture using a defined medium with specific inhibitor combinations. 

 

Inventor

Peter Klein, MD, PhD, Jian Huang, MD, PhD

 

Problem

Expanding hematopoietic stem cells (HSCs) is paramount in stem cell research and for clinical use of HSCs. However HSC homeostasis is not well understood and methods for HSC expansion have so far required the use of complex and expensive cocktails of cytokines and/or support cells. 

 

Solution

Dr. Klein and his team developed a method to expand and maintain HSCs ex-vivo using available

GSK3 and mTOR inhibitors potentially enabling production of a greater number of long term HSCs without the use of exogenous hematopoietic cytokines and other factors. This combination, in mice, increases the number of HSCs, improves the frequency of engraftment, and shortens the time to hematopoietic recovery in transplant recipients. Additionally, these novel culture conditions offer an excellent research tool to explore HSC biology.

  

Advantages

• Maintenance and expansion of HSCs without inducing cell lineage commitment

• Maintenance and expansion of HSCs without the need for cytokines

• Use of clinically approved drugs

 

 

Applications

• Pre-treatment to increase efficacy of bone marrow transplantation

• Pre-treatment to increase efficacy and survival rate of UCB transplantation

• Potential use to expand and maintain other stem cells

• Potential research tool to expand HSCs that may be applied to other adult stem cells 

 

Stage of Development

Ex-vivo maintemance of HSCs, including cord blood HSCs

In-vivo data showing improved survival of recipients who received HSCs cultured in inhibitor combination.

 

Intellectual Property

9,050,315

9,452,187

Pending US2017/0065640

 

Reference Media

Huang J et al. J Clin Invest, 2009, 119(12): 3519-29.

Huang J et al. Nat Med, 2012, 18(12): 1778-85.

 

Desired partnerships

• License

Co-development

  

Docket #  W5373

 


Patent Information:
For Information, Contact:
Viviane Martin
Director, PSOM Licensing Group
University of Pennsylvania
martinv@upenn.edu
Inventors:
Peter Klein
Jian Huang
Keywords: